Duchenne muscular dystrophy (DMD) is a progressive X-linked neuromuscular disorder affecting approximately 1 in 5,000 male births, causing deterioration of skeletal, cardiac, and respiratory muscle function.1 Corticosteroid therapy remains the cornerstone of management, with prednisone and deflazacort as standard of care.2 This narrative review compares prednisone, deflazacort, and vamorolone in pediatric DMD across four domains: motor function, linear growth, bone health, and overall safety. Semantic Scholar, PubMed, Embase, and ClinicalTrials.gov were searched for English-language studies (2000–2024) using terms including "Duchenne muscular dystrophy," "prednisone," "deflazacort," and "vamorolone," yielding approximately 1,100 records after deduplication. Titles and abstracts were screened against prespecified inclusion criteria (pediatric DMD, efficacy or safety outcomes), excluding case reports, editorials, and non-human studies. Fifty studies were selected by relevance ranking prioritizing RCTs, meta-analyses, and recency. Findings were organized thematically by the four predefined domains, with RCTs appraised using the Cochrane Risk of Bias tool.
Both prednisone and deflazacort preserve muscle strength and delay loss of ambulation by approximately 2–3 years, with deflazacort associated with less weight gain but potentially greater bone health impact.3,4 Both carry significant adverse effects including growth stunting, bone loss, adrenal suppression, and mood disturbances.5 Vamorolone at 6 mg/kg/day provides comparable efficacy to prednisone across TTSTAND, NSAA, and 6MWD, while preserving linear growth up to 30 months and improving bone biomarkers.6,7,8,9
Extending prior reviews by integrating 2024 trial data through a growth- and bone-health–focused lens, this synthesis positions vamorolone as a promising option for younger prepubertal patients, while acknowledging evidence-base limitations: short follow-up, no head-to-head deflazacort trials, and heterogeneous dosing regimens.
